Chang's team found that ASC-J9 breaks up the clumps by separating the androgen receptor from some of its helper molecules, or co-factors. As the clumps dissolve, CBP moves throughout the cell as it normally would, producing more VEGF.
Ultimately, the work brought about healthier mice whose symptoms were much improved. Chang's team also found that the amount of abnormal clumps in the neurons and spinal cords of the mice was slashed by half, and that VEGF activity in the neurons went up more than four times in the treated mice compared to untreated mice.
The new research is a signal that new avenues for treating conditions like prostate cancer, baldness and Kennedy's disease ?all of which rely on the androgen receptor ?are opening up, thanks to the dozens of molecular players that Chang and others have identified in the past decade.
"Traditionally, when scientists have wanted to affect the androgen receptor, they have taken away substances like testosterone that bind to and activate the receptor," said Chang. "But this can cause many unwanted, systemic side effects. We take another approach: Instead of taking away the substances that turn on the receptor, we look for ways to attack the faulty receptor directly. In this experiment, for instance, the mice treated with ASC-J9 have normal fertility and are completely healthy sexually, which wouldn't be the case if we simply took away testosterone."